Ignite Creation Date:
2025-12-24 @ 3:21 PM
Ignite Modification Date:
2026-02-09 @ 11:21 PM
Study NCT ID:
NCT02127892
Status:
TERMINATED
Last Update Posted:
2017-09-18
First Post:
2012-10-19
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
SCID Bu/Flu/ATG Study With T Cell Depletion
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D016511', 'term': 'Severe Combined Immunodeficiency'}], 'ancestors': [{'id': 'D000081207', 'term': 'Primary Immunodeficiency Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D007232', 'term': 'Infant, Newborn, Diseases'}, {'id': 'D049914', 'term': 'DNA Repair-Deficiency Disorders'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D007153', 'term': 'Immunologic Deficiency Syndromes'}, {'id': 'D007154', 'term': 'Immune System Diseases'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'nkapoor@chla.usc.edu', 'phone': '323-361-2546', 'title': 'Neena Kapoor, M.D.', 'organization': "Children's Hospital Los Angeles"}, 'certainAgreement': {'piSponsorEmployee': True}}, 'adverseEventsModule': {'timeFrame': 'Adverse event data was collected over a period of 1 year post transplant.', 'eventGroups': [{'id': 'EG000', 'title': 'Unrelated BM With T Cell Depletion', 'description': 'Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles.\n\nunrelated BM with T cell depletion: Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).', 'otherNumAtRisk': 7, 'deathsNumAtRisk': 7, 'otherNumAffected': 5, 'seriousNumAtRisk': 7, 'deathsNumAffected': 1, 'seriousNumAffected': 1}, {'id': 'EG001', 'title': 'Unrelated Cord Blood', 'description': 'Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles).\n\nunrelated cord blood: Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.', 'otherNumAtRisk': 2, 'deathsNumAtRisk': 2, 'otherNumAffected': 1, 'seriousNumAtRisk': 2, 'deathsNumAffected': 1, 'seriousNumAffected': 1}, {'id': 'EG002', 'title': 'Haplo BM With T Cell Depletion', 'description': 'If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors.\n\nhaplo BM with T cell depletion: haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.', 'otherNumAtRisk': 0, 'deathsNumAtRisk': 0, 'otherNumAffected': 0, 'seriousNumAtRisk': 0, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG003', 'title': 'Unrelated PBSC With T Cell Depletion', 'description': 'The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed.\n\nunrelated PBSC with T cell depletion: peripheral blood stem cell will be processed for CD34+ cell isolation.', 'otherNumAtRisk': 0, 'deathsNumAtRisk': 0, 'otherNumAffected': 0, 'seriousNumAtRisk': 0, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Graft Versus Host Disease', 'notes': "Graft versus host disease - condition post transplant the donor's immune cells in the transplant (graft) make antibodies against the patient's tissues (host) and attack vital organs (skin, GI track and liver common organs attacked)", 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numEvents': 5, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 0, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 0, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Infection - Aspergillis', 'notes': 'Pulmonary Aspergillis infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 0, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 0, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Infection - Fungal', 'notes': 'Pulmonary fungal infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 0, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 0, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Pulmonary Hemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 0, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 0, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Seizure', 'notes': 'Busulfan toxicity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 0, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 0, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'seriousEvents': [{'term': 'Sinusoidal obstruction syndrome', 'notes': 'Sinusoidal obstruction syndrome (SOS), previously known as veno-occlusive disease (VOD), is a distinctive and potentially fatal form of hepatic injury that occurs predominantly, if not only, after drug or toxin exposure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 7, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 0, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 0, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'SOS'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Number of Participants With Engraftment', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}, {'value': '0', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': 'Unrelated BM With T Cell Depletion', 'description': 'Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles.\n\nunrelated BM with T cell depletion: Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).'}, {'id': 'OG001', 'title': 'Unrelated Cord Blood', 'description': 'Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles).\n\nunrelated cord blood: Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.'}, {'id': 'OG002', 'title': 'Haplo BM With T Cell Depletion', 'description': 'If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors.\n\nhaplo BM with T cell depletion: haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.'}, {'id': 'OG003', 'title': 'Unrelated PBSC With T Cell Depletion', 'description': 'The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed.\n\nunrelated PBSC with T cell depletion: peripheral blood stem cell will be processed for CD34+ cell isolation.'}], 'classes': [{'categories': [{'measurements': [{'value': '7', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '100 day', 'description': 'Engraftment is defined as recovery of blood counts (neutrophil and platelet engraftment) with cells of donor origin, documented by either bone marrow or peripheral blood chimerism assays after hematopoietic stem cell transplant.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Number of Participants With Donor-derived CD3+ T Lymphocytes >/= 100/mm3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}, {'value': '0', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': 'Unrelated BM With T Cell Depletion', 'description': 'Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles.\n\nunrelated BM with T cell depletion: Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).'}, {'id': 'OG001', 'title': 'Unrelated Cord Blood', 'description': 'Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles).\n\nunrelated cord blood: Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.'}, {'id': 'OG002', 'title': 'Haplo BM With T Cell Depletion', 'description': 'If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors.\n\nhaplo BM with T cell depletion: haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.'}, {'id': 'OG003', 'title': 'Unrelated PBSC With T Cell Depletion', 'description': 'The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed.\n\nunrelated PBSC with T cell depletion: peripheral blood stem cell will be processed for CD34+ cell isolation.'}], 'classes': [{'categories': [{'measurements': [{'value': '7', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}, {'value': '0', 'groupId': 'OG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '1 year', 'description': 'Absolute number of donor-derived CD3+ T lymphocytes \\>/= 100/mm3 in participating subjects.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'Number of Participants With Veno-occlusive Disease (VOD) - Moderate and Severe', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}, {'value': '0', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': 'Unrelated BM With T Cell Depletion', 'description': 'Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles.\n\nunrelated BM with T cell depletion: Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).'}, {'id': 'OG001', 'title': 'Unrelated Cord Blood', 'description': 'Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles).\n\nunrelated cord blood: Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.'}, {'id': 'OG002', 'title': 'Haplo BM With T Cell Depletion', 'description': 'If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors.\n\nhaplo BM with T cell depletion: haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.'}, {'id': 'OG003', 'title': 'Unrelated PBSC With T Cell Depletion', 'description': 'The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed.\n\nunrelated PBSC with T cell depletion: peripheral blood stem cell will be processed for CD34+ cell isolation.'}], 'classes': [{'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}, {'value': '0', 'groupId': 'OG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '100 days', 'description': 'Evaluation of veno-occlusive disease determined by the presence of the following features; fluid retention, weight gain, leaky capillary syndrome, painful liver enlargement, refractoriness to platelet tranfusion and hyperbilirubinemia', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'Number of Participants With Graft Versus Host Disease (GVHD) - Grade III or IV', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}, {'value': '0', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': 'Unrelated BM With T Cell Depletion', 'description': 'Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles.\n\nunrelated BM with T cell depletion: Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).'}, {'id': 'OG001', 'title': 'Unrelated Cord Blood', 'description': 'Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles).\n\nunrelated cord blood: Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.'}, {'id': 'OG002', 'title': 'Haplo BM With T Cell Depletion', 'description': 'If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors.\n\nhaplo BM with T cell depletion: haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.'}, {'id': 'OG003', 'title': 'Unrelated PBSC With T Cell Depletion', 'description': 'The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed.\n\nunrelated PBSC with T cell depletion: peripheral blood stem cell will be processed for CD34+ cell isolation.'}], 'classes': [{'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}, {'value': '0', 'groupId': 'OG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '1 year', 'description': 'GVHD disease surveillance done by clinical evaluation, to include history, physical examination, specifically for rash, jaundice, liver dysfunction, nausea and vomiting, diarrhea and failure to thrive.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'Overall Survival', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}, {'value': '0', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': 'Unrelated BM With T Cell Depletion', 'description': 'Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles.\n\nunrelated BM with T cell depletion: Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).'}, {'id': 'OG001', 'title': 'Unrelated Cord Blood', 'description': 'Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles).\n\nunrelated cord blood: Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.'}, {'id': 'OG002', 'title': 'Haplo BM With T Cell Depletion', 'description': 'If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors.\n\nhaplo BM with T cell depletion: haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.'}, {'id': 'OG003', 'title': 'Unrelated PBSC With T Cell Depletion', 'description': 'The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed.\n\nunrelated PBSC with T cell depletion: peripheral blood stem cell will be processed for CD34+ cell isolation.'}], 'classes': [{'categories': [{'measurements': [{'value': '6', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}, {'value': '0', 'groupId': 'OG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '1 year', 'description': 'Overalls survival of patient at 1 year post transplant', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Unrelated BM With T Cell Depletion', 'description': 'Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles.\n\nunrelated BM with T cell depletion: Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).'}, {'id': 'FG001', 'title': 'Unrelated Cord Blood', 'description': 'Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles).\n\nunrelated cord blood: Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.'}, {'id': 'FG002', 'title': 'Haplo BM With T Cell Depletion', 'description': 'If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors.\n\nhaplo BM with T cell depletion: haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.'}, {'id': 'FG003', 'title': 'Unrelated PBSC With T Cell Depletion', 'description': 'The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed.\n\nunrelated PBSC with T cell depletion: peripheral blood stem cell will be processed for CD34+ cell isolation.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '7'}, {'groupId': 'FG001', 'numSubjects': '2'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '0'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '6'}, {'groupId': 'FG001', 'numSubjects': '1'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '0'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '1'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '0'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '9', 'groupId': 'BG004'}]}], 'groups': [{'id': 'BG000', 'title': 'Unrelated BM With T Cell Depletion', 'description': 'Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles.\n\nunrelated BM with T cell depletion: Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).'}, {'id': 'BG001', 'title': 'Unrelated Cord Blood', 'description': 'Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles).\n\nunrelated cord blood: Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.'}, {'id': 'BG002', 'title': 'Haplo BM With T Cell Depletion', 'description': 'If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors.\n\nhaplo BM with T cell depletion: haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.'}, {'id': 'BG003', 'title': 'Unrelated PBSC With T Cell Depletion', 'description': 'The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed.\n\nunrelated PBSC with T cell depletion: peripheral blood stem cell will be processed for CD34+ cell isolation.'}, {'id': 'BG004', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '7', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '9', 'groupId': 'BG004'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG004'}]}, {'title': 'Male', 'measurements': [{'value': '6', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '8', 'groupId': 'BG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '5', 'groupId': 'BG004'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '3', 'groupId': 'BG004'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '1', 'groupId': 'BG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG004'}]}, {'title': 'Asian', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG004'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG004'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG004'}]}, {'title': 'White', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG004'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG004'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '6', 'groupId': 'BG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '7', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '9', 'groupId': 'BG004'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 9}}, 'statusModule': {'whyStopped': 'Closed due to slow accrual. Nine subjects enrolled over 7 years.', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2007-01-02', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2017-08', 'completionDateStruct': {'date': '2016-08-01', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2017-08-18', 'studyFirstSubmitDate': '2012-10-19', 'resultsFirstSubmitDate': '2017-07-17', 'studyFirstSubmitQcDate': '2014-04-29', 'lastUpdatePostDateStruct': {'date': '2017-09-18', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2017-08-18', 'studyFirstPostDateStruct': {'date': '2014-05-01', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2017-09-18', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2016-08-01', 'type': 'ACTUAL'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Number of Participants With Veno-occlusive Disease (VOD) - Moderate and Severe', 'timeFrame': '100 days', 'description': 'Evaluation of veno-occlusive disease determined by the presence of the following features; fluid retention, weight gain, leaky capillary syndrome, painful liver enlargement, refractoriness to platelet tranfusion and hyperbilirubinemia'}, {'measure': 'Number of Participants With Graft Versus Host Disease (GVHD) - Grade III or IV', 'timeFrame': '1 year', 'description': 'GVHD disease surveillance done by clinical evaluation, to include history, physical examination, specifically for rash, jaundice, liver dysfunction, nausea and vomiting, diarrhea and failure to thrive.'}, {'measure': 'Overall Survival', 'timeFrame': '1 year', 'description': 'Overalls survival of patient at 1 year post transplant'}], 'primaryOutcomes': [{'measure': 'Number of Participants With Engraftment', 'timeFrame': '100 day', 'description': 'Engraftment is defined as recovery of blood counts (neutrophil and platelet engraftment) with cells of donor origin, documented by either bone marrow or peripheral blood chimerism assays after hematopoietic stem cell transplant.'}], 'secondaryOutcomes': [{'measure': 'Number of Participants With Donor-derived CD3+ T Lymphocytes >/= 100/mm3', 'timeFrame': '1 year', 'description': 'Absolute number of donor-derived CD3+ T lymphocytes \\>/= 100/mm3 in participating subjects.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isUnapprovedDevice': True, 'isFdaRegulatedDevice': True}, 'conditionsModule': {'keywords': ['Severe Combined Immune Deficiency', 'SCID', 'HLA-matched unrelated donor', 'unrelated', 'MUD', 'bone marrow', 'cord blood', 'haplo-identical', 'peripheral blood', 'CD34+ selection', 'T cell depletion', 'HSCT', 'BMT', 'CliniMACS'], 'conditions': ['Severe Combined Immunodeficiency']}, 'descriptionModule': {'briefSummary': 'This is a pilot clinical trial of hematopoietic stem cell transplantation for patients with a diagnosis of Severe Combined Immune Deficiency (SCID) who do not have an HLA-matched sibling donor. The stem cells will be derived from a 1) matched unrelated donor (MUD), 2) unrelated cord blood donor, or 3) a haplo-identical (parental) donor (in descending order of preference).Patients will receive a novel conditioning regimen with Busulfan, Fludarabine and Anti-thymocyte globulin (ATG) followed by an unrelated donor hematopoietic stem cell transplant (HSCT) with T-cell depletion using the CliniMACS device.', 'detailedDescription': 'The study is being conducted to assess the following:\n\n* overall survival\n* event-free survival (events are defined as: death,non-engraftment/2nd transplant, immune reconstitution failure)\n* acute toxicity of the conditioning regimen\n* engraftment frequency immune reconstitution frequency and tempo acute and chronic graft-versus-host disease (GVHD), frequency and severity.\n\nThe outcome from this protocol will be compared to the retrospective cohort consisting of all patients who have undergone haplo-identical HSCT for SCID at CHLA from 1984-2006 based on the assessment of the above-listed endpoints.\n\nThe CliniMACS device will be used for CD34+ selection in place of the Isolex 300i. The CliniMACS CD34 Reagent System is an investigational medical device that has not yet been approved by the FDA. This device is used in vitro to select and enrich specific cell populations. When using the CliniMACS CD34 Reagent, the system selects CD34+ cells from heterogenous hematological cell populations for transplantation in cases where this is clinically indicated.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '21 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* All patients with SCID who lack a histocompatible sibling or HLA-matched related donor will be considered as candidates for this study protocol.\n* Eligible patients must have adequate physical function to tolerate the chemotherapy conditioning regimen and the HSCT, as measure by:\n\n 1. Renal: creatinine clearance or GFR ≥50 ml/min/1.73m2, and not requiring dialysis\n 2. Pulmonary: Because patients with SCID frequently present with infectious pneumonia causing ventilatory failure, patients will be considered for enrollment in the study even if respiratory failure requiring mechanical ventilatory support is present. In patients recently diagnosed with pneumonia, efforts to stabilize the respiratory status will be made prior to enrollment in the study.\n 3. Infectious disease status. The presence of infection per se will not be a reason for exclusion from the study. Patients with SCID are frequently infected with both routine pathogens as well as opportunistic infections. Antibiotic, antifungal and antiviral prophylaxis and therapy will be instituted as clinically indicated. Despite the use of antimicrobial therapy, the ability to control infections will not be achieved unless HSCT is performed. Therefore, subjects may be enrolled in the study, even though infection is present, because control of infection may depend on engraftment of a donor immune system.\n 4. Patients will be 0-21 years of age.\n\nExclusion Criteria:\n\n* Patient with histocompatible sibling or other related donor\n* End-organ failure that precludes the ability to tolerate the transplant procedure, including conditioning.\n* Renal failure requiring dialysis\n* Congenital heart disease resulting in congestive heart failure\n* Severe CNS disease, e.g., coma or intractable seizures\n* Ventilatory failure due to non-infectious etiology\n* Major congenital anomalies that adversely affect survival, eg CNS malformations\n* Metabolic diseases that would affect transplant survival, eg urea cycle disorders\n* HIV infection\n\nSince the only chance of survival for patients with SCID is successful transplantation, all patients with SCID will be considered to be potential subjects for the study, regardless of end-organ dysfunction.'}, 'identificationModule': {'nctId': 'NCT02127892', 'briefTitle': 'SCID Bu/Flu/ATG Study With T Cell Depletion', 'organization': {'class': 'OTHER', 'fullName': "Children's Hospital Los Angeles"}, 'officialTitle': 'Phase I/II Trial of Hematopoietic Stem Cell Transplant (HSCT) for Children With Severe Combined Immune Deficiency (SCID) and Without an HLA-Matched Sibling Donor', 'orgStudyIdInfo': {'id': 'CCI-06-00243'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'OTHER', 'label': 'unrelated BM with T cell depletion', 'description': 'Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles.', 'interventionNames': ['Biological: unrelated BM with T cell depletion']}, {'type': 'OTHER', 'label': 'unrelated cord blood', 'description': 'Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles).', 'interventionNames': ['Biological: unrelated cord blood']}, {'type': 'OTHER', 'label': 'haplo BM with T cell depletion', 'description': 'If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors.', 'interventionNames': ['Biological: haplo BM with T cell depletion']}, {'type': 'OTHER', 'label': 'unrelated PBSC with T cell depletion', 'description': 'The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed.', 'interventionNames': ['Device: unrelated PBSC with T cell depletion']}], 'interventions': [{'name': 'unrelated BM with T cell depletion', 'type': 'BIOLOGICAL', 'otherNames': ['CD34+ selection using CliniMACS'], 'description': 'Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).', 'armGroupLabels': ['unrelated BM with T cell depletion']}, {'name': 'unrelated cord blood', 'type': 'BIOLOGICAL', 'otherNames': ['umbilical cord blood'], 'description': 'Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.', 'armGroupLabels': ['unrelated cord blood']}, {'name': 'haplo BM with T cell depletion', 'type': 'BIOLOGICAL', 'otherNames': ['CD34+ selection with CliniMACS'], 'description': 'haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.', 'armGroupLabels': ['haplo BM with T cell depletion']}, {'name': 'unrelated PBSC with T cell depletion', 'type': 'DEVICE', 'otherNames': ['CD34+ selection using CliniMACS'], 'description': 'peripheral blood stem cell will be processed for CD34+ cell isolation.', 'armGroupLabels': ['unrelated PBSC with T cell depletion']}]}, 'contactsLocationsModule': {'locations': [{'zip': '90027', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': "Children's Hospital Los Angeles", 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}], 'overallOfficials': [{'name': 'Neena Kapoor, M.D.', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': "Children's Hospital Los Angeles, University of Southern California"}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Neena Kapoor, M.D.', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR_INVESTIGATOR', 'investigatorTitle': 'Professor of Pediatrics, Keck School of Medicine; Division Head, Division of Research Immunology/BMT', 'investigatorFullName': 'Neena Kapoor, M.D.', 'investigatorAffiliation': "Children's Hospital Los Angeles"}}}}